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FOR-DMD: Recruitment and Retention Challenges

FOR-DMD: Recruitment and Retention Challenges. Addressing the Challenges Benefits vs risks of corticosteroids? Why invest so much to study a treatment of established (short term) benefit? Corticosteroids vs curative new treatment? Does FOR-DMD preclude participation in another study?

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FOR-DMD: Recruitment and Retention Challenges

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  1. FOR-DMD: Recruitment and Retention Challenges Addressing the Challenges Benefits vs risks of corticosteroids? Why invest so much to study a treatment of established (short term) benefit? Corticosteroids vs curative new treatment? Does FOR-DMD preclude participation in another study? Can subjects leave FOR-DMD to enter another study?

  2. Benefits vs risks of corticosteroids? • No longer equipoise concerning corticosteroids vs placebo (study sections X3; FOR-DMD investigators; CINRG Network; Cochrane, AAN reviews) • Long-term data, albeit uncontrolled • New trials are “permitting” corticosteroids

  3. Why invest so much to study a treatment of established (short term) benefit? • No standard treatment (31 regimens) • No standards of side effect prevention/long-term treatment of DMD • Heterogeneity of regimens may defeat and unquestionably complicates new treatment evaluations (e.g., PTC 124)

  4. Heterogeneous Steroid Regimens: A Challenge for New Therapeutic Approaches • Different benefits from different agents? • Varying time courses of benefit • Improved in 10-90 days • Plateau in 3-12 mos • Decline in 18-? mos • Worsen off treatment in ? • Effect of variation on study power • Adverse effects differ • Different drug interactions.

  5. Corticosteroids vs curative new treatment? • No proposed new treatment likely curative • Few new treatments applicable to all DMD • People underestimate the benefit of corticosteroids • 30% longer ambulation • 30% longer off ventilator • Survive? • (Better than pacemakers, defibrillators, all cancer chemotherapy

  6. Does FOR-DMD preclude participation in another study? Yes Can subjects leave FOR-DMD to enter another study? Yes

  7. This trial will also establish standards of care in 41 centers and 11 countries.

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